MONTREAL, Aug. 25, 2015 /PRNewswire/ -- Clementia Pharmaceuticals, Inc. today announced the initiation of enrollment of children as young as 6 years old in the company's ongoing Phase 2 study of palovarotene for the treatment of fibrodysplasia ossificans progressiva (FOP). The company also announced the addition of a clinical trial site in the United Kingdom that should ease the travel burden for affected individuals living in the European Union. FOP is an extremely rare and severely disabling congenital myopathy characterized by heterotopic ossification (HO), the abnormal growth of bone in muscles, tendons and ligaments, causing significant morbidities and progressive disability. There are currently no approved treatments for FOP.
Clementia expanded enrollment to include children with FOP in the ongoing Phase 2 clinical trial following completion of the appropriate nonclinical studies. In addition to the 24 patients already enrolled in this adaptive-design, dose-ranging study, an additional 16 patients will be enrolled, with at least eight being between the ages of 6 and 14. The company anticipates completion of enrollment by the end of 2015.
"The inclusion of children as young as 6 years old in the ongoing Phase 2 clinical trial will enable a preliminary evaluation of the safety and efficacy of palovarotene in a younger population, a particularly important consideration given the early onset of FOP flare-ups," commented Donna Grogan, M.D., Chief Medical Officer of Clementia. "Once the Phase 2 study is complete, we will analyze the data as quickly as possible to inform the design of our planned Phase 3 study, which we anticipate starting in the second half of 2016."
Clementia also announced the opening of a fourth clinical trial site at Royal National Orthopaedic Hospital in London, United Kingdom for the ongoing Phase 2 clinical trial, with Dr. Richard Keen as the principal investigator.
"We are pleased to participate in this important research to further serve patients living in Europe," noted Dr. Richard Keen, Royal National Orthopaedic Hospital. Chris Bedford-Gay, father of a young child with FOP and founder of FOP Friends® the UK Charity and FOP support group, commented, "We appreciate Clementia's commitment in accelerating the development of a potential treatment for FOP, one that cannot come quickly enough to families like ours."
At this time, individuals with FOP age 15 or older can enroll at the UK site as well as all other sites, which include the University of California San Francisco (UCSF), University of Pennsylvania in Philadelphia, and Hopital Necker-Enfants Malades in Paris, France. UCSF is the first site to open enrollment to children with FOP age 6 and older who meet eligibility criteria. All other sites plan to initiate enrollment of younger children as they receive approvals from their respective institutions.
About Fibrodysplasia Ossificans Progressiva (FOP) FOP is a rare, severely disabling congenital myopathy characterized by heterotopic ossification (HO) of muscles, tendons, and ligaments. Soft tissue swellings called flare-ups often precede heterotopic bone formation. Flare-ups typically begin early in life and may occur spontaneously or after soft tissue trauma, muscular stretching, over exertion or fatigue, intramuscular vaccinations, mandibular blocks (nerve block injection) for dental work, or flu-like infections. Episodes of heterotopic bone formation progressively restrict movement by locking joints, leading to cumulative loss of function, disability, and death. Nearly 100 percent of newborns with FOP have a hallmark toe malformation in which the big toes are shortened and bent inwards. FOP is caused by a mutation in the ACVR1 gene resulting in increased activity of the activin receptor type I (ACVR1/ALK2) involved in the bone morphogenic protein (BMP) pathway, a key pathway controlling bone growth and development. There are currently no approved treatments for FOP.
About Palovarotene Palovarotene is a retinoic acid receptor gamma agonist being investigated as a treatment for FOP. Preclinical studies demonstrated that palovarotene blocked abnormal bone formation in animal models. Clementia inlicensed palovarotene from Roche Pharmaceuticals, which previously evaluated the compound in more than 800 individuals including healthy volunteers and patients with chronic obstructive pulmonary disease. Palovarotene received Fast Track designation from the U.S. Food and Drug Administration (FDA) and orphan designations for the treatment of FOP from both the FDA and the European Medicines Agency (EMA).
About the Clinical Trial The double-blind, placebo-controlled Phase 2 clinical trial is evaluating the effect of different doses of palovarotene on new bone formation during and after a flare-up in 40 patients with FOP who are 6 years of age or older. In the trial, treatment is initiated within seven (7) days from flare-up onset and continued for six (6) weeks with an additional six (6) weeks of follow-up. Efficacy and safety endpoints include imaging endpoints for new bone formation, clinical assessments of physical function and patient-reported outcomes. In preclinical studies, palovarotene prevented heterotopic bone formation in mouse models of FOP. The ongoing Phase 2 clinical trial was designed to determine whether these effects can be replicated in patients with FOP.
About Clementia Pharmaceuticals Inc. Clementia is a clinical-stage biopharmaceutical company committed to delivering treatments to those who have none. The company is developing its lead candidate palovarotene, a novel retinoic acid receptor gamma agonist, to treat fibrodysplasia ossificans progressiva (FOP) and other diseases. For more information, please visit www.clementiapharma.com.